Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome:: collaborative study of the International Bone Marrow Transplant registry and the National Marrow Donor Program
Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome:: collaborative study of the International Bone Marrow Transplant registry and the National Marrow Donor Program
复制标题
DOI：
10.1182/blood.v97.6.1598
复制
发表时间：
2001-03-15
期刊：
影响因子：
20.3
通讯作者：
Horowitz, MM
中科院分区：
文献类型：
--
作者：
Filipovich, AH;Stone, JV;Horowitz, MM
人类白细胞抗原（HLA）相合同胞骨髓移植是治疗Wiskott-Aldrich综合征的有效方法。然而，大多数患有这种疾病的儿童缺乏这样的供体，许多患者接受来自替代供体的移植。本研究比较了HLA相合同胞、其他亲缘供者和非亲缘供者移植治疗Wiskott-Aldrich综合征的结果。对1968年至1996年国际骨髓移植登记处和/或国家骨髓捐献者计划报告的170例Wiskott-Aldrich综合征移植的结果进行了评估。55例来自HLA相同的同胞供体，48例来自其他亲属，67例来自无关供体。多变量比例风险回归用于比较供体类型的结果，并确定其他预后因素。大多数移植受者年龄小于5岁（79%），移植前表现评分大于或等于90%（63%），接受了无放射的移植前准备方案（82%），并且具有非T细胞耗尽的移植物（77%），80%在1986年之后接受移植，5年生存概率所有受试者的概率（95%置信区间）为70%（63%-77%），不同供体类型的概率不同：87%（74%-93%）与HLA相同的同胞供体，52%（37%-65%）与其他相关供体，71%（58%-80%）与无关供体（P = 0.0006）。多变量分析表明，与HLA相同的同胞移植相比，使用HLA相同同胞以外的相关供体（P = .0004）或5岁以上男孩的无关供体（P = .0001）的存活率显著降低。5岁之前接受无关供体移植的男孩的存活率与接受HLA相同的同胞移植的男孩相似。Wiskott-Aldrich综合征的最佳移植结局是使用HLA相同的同胞供体。在年幼的儿童中，无关供体的存活率可能相当。（血。2001;97：1598-1603）（C）2001年由美国血液学会。
Human leukocyte antigen (HLA)-identical sibling bone marrow transplantation is an effective treatment for Wiskott-Aldrich syndrome. However, most children with this disease lack such donors and many patients receive transplants from alternative donors. This study compared outcomes of HLA-identical sibling, other related donor, and unrelated donor transplantation for Wiskott-Aldrich syndrome. The outcome of 170 transplantations for Wiskott-Aldrich syndrome, from 1968 to 1996, reported to the International Bone Marrow Transplant Registry and/or National Marrow Donor Program were assessed. Fifty-five were from HLA-identical sibling donors, 48 from other relatives, and 67 from unrelated donors. Multivariate proportional hazards regression was used to compare outcome by donor type and identify other prognostic factors. Most transplant recipients were younger than 5 years (79%), had a pretransplantation performance score greater than or equal to 90% (63%), received pretransplantation preparative regimens without radiation (82%), and had non-T-cell-depleted grafts (77%), Eighty percent received their transplant after 1986, The 5-year probability of survival (95% confidence interval) for all subjects was 70% (63%-77%), Probabilities differed by donor type: 87% (74%-93%) with HLA-identical sibling donors, 52% (37%-65%) with other related donors, and 71% (58%-80%) with unrelated donors (P = .0006). Multivariate analysis indicated significantly lower survival using related donors other than HLA-identical siblings (P = .0004) or unrelated donors in boys older than 5 years (P = .0001), compared to HLA-identical sibling transplants. Boys receiving an unrelated donor transplant before age 5 had survivals similar to those receiving HLA-identical sibling transplants. The best transplantation outcomes in Wiskott-Aldrich syndrome are achieved with HLA-identical sibling donors. Equivalent survivals are possible with unrelated donors in young children. (Blood. 2001;97:1598-1603) (C) 2001 by The American Society of Hematology.